Human medicines European public assessment report (EPAR): Lacosamide Adroiq, lacosamide, Date of authorisation: 31/05/2023, Revision: 5, Status: Authorised

Orphan designation: Fosmanogepix Treatment of fusariosis, 15/02/2023 Positive

Orphan designation: Heterologous human adult liver derived  stem cells Treatment of ornithinine transcarbamylase deficiency, 04/02/2008 Positive

Orphan designation: Heterologous human adult liver-derived progenitor cells Treatment of citrullinaemia type 1, 17/07/2013 Positive

Orphan designation: Heterologous human adult liver-derived stem cells Treatment of Crigler-Najjar syndrome, 29/11/2007 Positive

Orphan designation: Heterologous human adult liver-derived progenitor cells Treatment of hyperargininaemia, 17/07/2013 Positive

Orphan designation: Heterologous human adult liver-derived progenitor cells Treatment of carbamoyl-phosphate synthase-1 deficiency, 17/07/2013 Positive

Orphan designation: Heterologous human adult liver-derived progenitor cells Treatment of citrullinaemia type 2, 17/08/2013 Positive

Orphan designation: Heterologous human adult liver-derived progenitor cells Treatment of N-acetylglutamate synthetase (NAGS) deficiency, 17/07/2013 Positive

Human medicines European public assessment report (EPAR): Siklos, hydroxycarbamide, Date of authorisation: 29/06/2007, Revision: 26, Status: Authorised